TY - JOUR
T1 - Thrombotic Thrombocytopenic Purpura and Evans Syndrome
T2 - Validating and Exploring 20 Years of Routine Hospital Care
AU - Lawrie, Dana Audrey
AU - Hansen, Dennis Lund
AU - Kristensen, Thomas Leineweber
AU - Carlsen, Sarah Birgitte Ingemod Sand
AU - Hannig, Louise Hur
AU - Pedersen, Per Trøllund
AU - Kristensen, Helene Bjørg
AU - Lorenzen, Mads Okkels Birk
AU - Stentoft, Jesper
AU - Van Kooten Niekerk, Peter Buur
AU - Jørgensen, Maren Poulsgaard
AU - Severinsen, Marianne Tang
AU - Dorff, Mikkel Helleberg
AU - Pedersen, Robert Schou
AU - Glenthøj, Andreas
AU - Frederiksen, Henrik
N1 - © 2025 Lawrie et al.
PY - 2025
Y1 - 2025
N2 - PURPOSE: Few patients scattered among centers complicate investigation of thrombotic thrombocytopenic purpura (TTP) and Evans syndrome (ES). Routinely collected Danish register data captures the total population and includes lifelong follow-up. We aimed to validate registered TTP and ES diagnoses and to explore clinical characteristics.PATIENTS AND METHODS: We identified all patients in Denmark with diagnosis registrations indicative of TTP or ES in the Danish National Patient Registry 2000-2019, validated diagnoses through medical record review, and extracted and presented data on initial treatment and complications.RESULTS: Diagnoses for patients registered with TTP and ES were confirmed for 46% and 59%, respectively. Among validated TTP patients the most widespread complications at time of diagnosis were neurological symptoms or deficits, observed in 81% of cases. Other frequent types of complications in TTP patients were any organ failure (32%) and infection (25%). Initial management and complications did not change for patients diagnosed between 2000 and 2009 and 2010 and 2019, and survival remained constant (overall mortality 26%, median follow up of 8.4 years). Treatments and complications also remained unchanged for ES patients.CONCLUSION: Overall, diagnostic accuracy, complications and prognosis have remained relatively constant for patients over the study period. These now validated cohorts of Danish TTP and ES patients will be utilized in future studies to examine long-term health outcomes.
AB - PURPOSE: Few patients scattered among centers complicate investigation of thrombotic thrombocytopenic purpura (TTP) and Evans syndrome (ES). Routinely collected Danish register data captures the total population and includes lifelong follow-up. We aimed to validate registered TTP and ES diagnoses and to explore clinical characteristics.PATIENTS AND METHODS: We identified all patients in Denmark with diagnosis registrations indicative of TTP or ES in the Danish National Patient Registry 2000-2019, validated diagnoses through medical record review, and extracted and presented data on initial treatment and complications.RESULTS: Diagnoses for patients registered with TTP and ES were confirmed for 46% and 59%, respectively. Among validated TTP patients the most widespread complications at time of diagnosis were neurological symptoms or deficits, observed in 81% of cases. Other frequent types of complications in TTP patients were any organ failure (32%) and infection (25%). Initial management and complications did not change for patients diagnosed between 2000 and 2009 and 2010 and 2019, and survival remained constant (overall mortality 26%, median follow up of 8.4 years). Treatments and complications also remained unchanged for ES patients.CONCLUSION: Overall, diagnostic accuracy, complications and prognosis have remained relatively constant for patients over the study period. These now validated cohorts of Danish TTP and ES patients will be utilized in future studies to examine long-term health outcomes.
KW - ADAMTS13
KW - TTP
KW - thrombotic microangiopathy
KW - validation
UR - https://www.scopus.com/pages/publications/105008229773
U2 - 10.2147/JBM.S513578
DO - 10.2147/JBM.S513578
M3 - Journal article
C2 - 40502537
SN - 1179-2736
VL - 16
SP - 279
EP - 292
JO - Journal of Blood Medicine
JF - Journal of Blood Medicine
ER -